Our Science

We own a proprietary platform technology targeting aging, inflammation and senescence, focused at delivering a therapeutic gene that has demonstrated strong clinical efficacy and safety in treating cardiovascular, pulmonary and orthopedic conditions.

We have recently reached an exclusive license agreement with the Icahn School of Medicine at Mount Sinai in New York, NY to a broad patent portfolio related to Sphingolipids metabolizing enzyme (SME) and its therapeutic application in senescence, inflammation and aging. This high value technology has been developed in the lab of Efrat Eliyahu, PhD, Assistant Professor of Genetics and Genomics Sciences at Icahn Mount Sinai, and is addressing acute and chronic inflammatory diseases and related conditions. The therapeutic protein is administered as gene therapy via using adeno-associated-virus mediated delivery. Icahn Mount Sinai will continue to partner with Gromit to support Gromit-funded research and clinical efforts that leverage Icahn Mount Sinai’s extraordinary expertise and facilities.

Our technology is also supported by multiple peer reviewed publications, clinical and scientific collaborations and NIH grants.

Selected Publications

1. Katz, M. G., Hadas, Y., Bailey, R. A., Fazal, S., Vincek, A., Madjarova, S. J., Shtraizent, N., Vandenberghe, L. H., & Eliyahu, E. (2021). Efficient Cardiac Gene Transfer and Early Onset Expression of a Synthetic Adeno-Associated Viral Vector, Anc80L65 After Intramyocardial Administration. The Journal of Thoracic and Cardiovascular Surgery. https://doi.org/10.1016/j.jtcvs.2021.05.050 

2. Orlowski, A., Katz, M. G., Gubara, S. M., Fargnoli, A. S., Fish, K. M., & Weber, T. (2020). Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption. Molecular Therapy - Methods & Clinical Development, 16, 192–203. https://doi.org/10.1016/j.omtm.2020.01.004 

3. el Andari, J., & Grimm, D. (2020). Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors. Biotechnology Journal, 16(1), 2000025. https://doi.org/10.1002/biot.202000025 

4. Katz, M. G., Gubara, S. M., Hadas, Y., Weber, T., Kumar, A., Eliyahu, E., Bridges, C. R., & Fargnoli, A. S. (2020). Effects of genetic transfection on calcium cycling pathways mediated by double-stranded adeno-associated virus in postinfarction remodeling. The Journal of Thoracic and Cardiovascular Surgery, 159(5), 1809–1819.e3. https://doi.org/10.1016/j.jtcvs.2019.08.089 

5. Katz, M. G., Gubara, S. M., & Eliyahu, E. (2020). Gene therapy targets in postinfarction remodeling. The Journal of Thoracic and Cardiovascular Surgery, 159(5), e305. https://doi.org/10.1016/j.jtcvs.2019.11.122 

6. Hadas, Y., Vincek, A. S., Youssef, E., ŻAk, M. M., Chepurko, E., Sultana, N., Sharkar, M. T. K., Guo, N., Komargodski, R., Kurian, A. A., Kaur, K., Magadum, A., Fargnoli, A., Katz, M. G., Hossain, N., Kenigsberg, E., Dubois, N. C., Schadt, E., Hajjar, R., . . . Zangi, L. (2020). Altering Sphingolipid Metabolism Attenuates Cell Death and Inflammatory Response After Myocardial Infarction. Circulation, 141(11), 916–930. https://doi.org/10.1161/circulationaha.119.041882 

7. Ikeda, Y., Sun, Z., Ru, X., Vandenberghe, L. H., & Humphreys, B. D. (2018). Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector. Journal of the American Society of Nephrology, 29(9), 2287–2297. https://doi.org/10.1681/asn.2018040426 

8. Simonaro, C. M., Sachot, S., Ge, Y., He, X., DeAngelis, V. A., Eliyahu, E., Leong, D. J., Sun, H. B., Mason, J. B., Haskins, M. E., Richardson, D. W., & Schuchman, E. H. (2013). Acid Ceramidase Maintains the Chondrogenic Phenotype of Expanded Primary Chondrocytes and Improves the Chondrogenic Differentiation of Bone Marrow-Derived Mesenchymal Stem Cells. PLoS ONE, 8(4), e62715. https://doi.org/10.1371/journal.pone.0062715 

9. Eliyahu, E., Shtraizent, N., Shalgi, R., & Schuchman, E. H. (2012). Construction of Conditional Acid Ceramidase Knockout Mice andin vivoEffects on Oocyte Development and Fertility. Cellular Physiology and Biochemistry, 30(3), 735–748. https://doi.org/10.1159/000341453

10. Eliyahu, E., Wolfson, T., Ge, Y., Jepsen, K. J., Schuchman, E. H., & Simonaro, C. M. (2011). Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI. PLoS ONE, 6(8), e22447. https://doi.org/10.1371/journal.pone.0022447 

11. Eliyahu, E., Shtraizent, N., Martinuzzi, K., Barritt, J., He, X., Wei, H., Chaubal, S., Copperman, A. B., & Schuchman, E. H. (2009). Acid ceramidase improves the quality of oocytes and embryos and the outcome of in vitro fertilization. The FASEB Journal, 24(4), 1229–1238. https://doi.org/10.1096/fj.09-145508 

12. Eliyahu, E., Park, J. H., Shtraizent, N., He, X., & Schuchman, E. H. (2007). Acid ceramidase is a novel factor required for early embryo survival. The FASEB Journal, 21(7), 1403–1409. https://doi.org/10.1096/fj.06-7016com